Genetix Biotherapeutics

bluebird bio (formerly Genetix Biotherapeutics) is a commercial-stage biotechnology company that develops transformative gene therapies for severe genetic diseases. The company utilizes a proprietary lentiviral vector platform to modify a patient's own hematopoietic stem cells ex vivo, aiming to provide a one-time curative treatment. Its FDA-approved portfolio includes Zynteglo for transfusion-dependent beta-thalassemia, Skysona for cerebral adrenoleukodystrophy, and Lyfgenia for sickle cell disease. Founded in 1992 and rebranded in 2010, the company is a pioneer in the field of gene therapy. In 2021, bluebird bio spun off its oncology-focused programs into a separate entity, 2seventy bio, to focus exclusively on severe genetic diseases.